Ophthotech Nabs $175M To Fund Late-Stage Trial For Eye Drug

5/29/13Follow @benthefidler

Ophthotech was founded by former Eyetech Pharmaceuticals executives to change the standard of care for the “wet” form of age-related macular degeneration: the leading cause of blindness among adults in the western world.

With a new $175 million round of financing the New York company will get its shot to prove its new drug candidate works in a large-scale clinical trial.

New York-based Ophthotech announced today that is has raised the new round of cash from Novo A/S and its current investors, which include SV Life Sciences, Novo Ventures (the VC arm of Novo Nordisk), HBM Healthcare Investments, and Clarus Ventures.

The financing comes in a couple parts. One is a $50 million Series C round of preferred equity financing, which is being supplied by both Novo, and Ophthotech’s other investors. The remaining $125 million is being committed by Novo Nordisk as part of a product royalty sale agreement. Essentially, Ophthotech is providing Novo with an undisclosed percentage royalty on future sales of its drug candidate E10030 (which it hopes to market under the name Fostiva). In return, Novo has agreed to pay $125 million in three equal installments. The first part of the Series C and the royalty financing deal has already closed, which means that Ophthotech should have about $58.3 million of new cash for its coffers today, and that it stands to collect roughly another $116.7 million from Novo and its other investors if its drug can hit all of its milestones.

Ophthotech will use the cash to bankroll a large late-stage study of E10030, a treatment for the wet form of AMD, a condition in which blood vessels leak behind the eye, leading to distorted vision and potentially blindness. Ophthotech hasn’t revealed the design of the study yet, but it will include 1,900 patients across 200 testing sites, and begin before the end of September.

The market for age-related macular degeneration has exploded over the past several years, as the standard of care has changed from laser therapies that didn’t actually improve patients’ vision—rather, it stopped their vision from getting worse while destroying the very retinal tissue clinicians were trying to save—to injectable pharmaceuticals that do. Roche/Genentech’s ranibizumab (Lucentis) and bevacizumab (Avastin), and Regeneron Pharmaceuticals’ afilbercept (Eylea) have all become hit drugs for treating the disease. All of them, however, fight AMD by inhibiting the vascular endothelial growth factor (VEGF) that researchers say causes the condition to worsen. Ophthotech’s E10030, instead, blocks platelet-derived growth factor (PDGF), a different protein that has also been implicated in the wet form of AMD.

This is important because the essential problem in the wet form of AMD is abnormal blood vessel growth in the macula, a part of the retina. While blocking VEGF on its own only stops those abnormal blood vessels from continuing to grow, scientists believe adding PDGF to the equation may cause them to recede. Ophthotech’s plan is to combine the two treatments into one regimen, giving patients one injection of an anti-VEGF such as ranibizumab, and another of E10030. While the idea of getting two injections in the eye in one sitting certainly isn’t a fun idea for potential patients, Ophthotech’s selling point is that combining the two simply works better than solely using any anti-VEGF on its own.

“Giving combination therapy via two injections does not increase the treatment burden for the patient or physician,” says Ophthotech CEO David Guyer. “The patient visits are unchanged.”

Ophthotech isn’t the only company to target PDGF—a startup in Waltham, MA called Kala Pharmaceuticals is developing a drug, for example, that blocks both VEGF and PDGF—but Ophthotech is the farthest along in development. The company in 2012 completed a mid-stage study of … Next Page »

Ben Fidler is Xconomy's Deputy Biotechnology Editor. You can e-mail him at bfidler@xconomy.com Follow @benthefidler

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